Denali Therapeutics is committed to discovering, developing and delivering medicines for people with neurodegenerative diseases, lysosomal storage disorders and other serious diseases. We recognize the urgency faced by individuals and families affected by these conditions and are grateful to those who participate in our clinical studies. Clinical studies are essential to evaluate the safety and effectiveness of investigational therapies and generate the evidence needed to bring new medicines to patients as quickly as possible.

Expanded access, also known as compassionate use, is a form of early access that may allow patients with serious or life-threatening conditions to access investigational medicines before they are approved by a regulatory authority such as the FDA or EMA. Currently, Denali Therapeutics does not offer expanded access to investigational treatments.

Denali Therapeutics believes that the best way to make medicines available to the patient community is through well-designed clinical studies followed by approval by regulatory authorities. At the same time, we understand that not all patients are able to participate in a clinical study or access medicines that have received regulatory approval.

We recognize the urgent need for treatment options. At Denali, we are doing all we can to complete clinical studies that are both robust and expeditious to secure regulatory approval and access. As more clinical data on the safety and efficacy of our investigational medicines become available, we will review and potentially revise our expanded access policy.

AVLAYAH (tividenofusp alfa) was granted accelerated approval by the United States FDA in March 2026 for the treatment of neurological manifestations of Hunter syndrome (mucopolysaccharidosis type II, MPS II) when initiated in presymptomatic or symptomatic pediatric patients weighing at least 5 kg prior to advanced neurological impairment. At this time, we do not offer expanded access for AVLAYAH (tividenofusp alfa). In select countries outside the United States, the treating physician may request early access to tividenofusp alfa via country-specific pathways and in line with local regulatory requirements. Physicians outside the United States seeking more information about early access on behalf of a patient may contact Denali Therapeutics via the website’s General Contact form and select “Medical Affairs” in the Department field.